Nov 22, 2017

The Value of iPSCs for Stem Cell Therapy

Stem cell therapy has emerged as one of the most promising approaches in the fight to end blindness, and Dr. Gilbert Bernier’s research has played an enormous role in pushing the field forward.

By developing new techniques to turn induced pluripotent stem cells (iPSCs) into sheets of cone photoreceptors, Dr. Bernier and his team are bringing us closer than ever to the transplantation of those cells into the eyes of those living with blinding eye diseases such as Stargardt Disease and retinitis pigmentosa. The goal is not just to prevent the further loss of sight in these individuals, but to restore sight, to give them their vision back.

Scientists conduct research with a range of different types of stem cells, and those cells can be found in different places throughout the adult body and during development. The incredible thing about iPSCs is that they can be derived from adult skin cells. They can also be produced in a patient-specific manner, meaning that they can be tailored to match the biology of the donor in a way that will avoid the risk of immune system rejection.

Dr. Bernier’s use of iPSCs is ground-breaking in two very specific ways.

First, he and his team are using the cells to produce sheets of functioning cone photoreceptors in vitro (in a dish)—photoreceptors are arranged in “sheets,” flat surfaces ideal for absorbing light particles. This is essential: if the goal is to transplant photoreceptors into the eyes of patients living with vision loss, we must first ensure that we have a safe and reliable way of producing those cells outside of the eye. If we do not, the cells that are transplanted could be faulty and could lead to any number of issues.

The work that Dr. Bernier and his team are doing in this area is cutting edge, and is built off of their extraordinary discovery in 2015 of a new protein, COCO, responsible for nudging stem cells into cone photoreceptors, the cells responsible for central vision. In the two years since that discovery they have made significant progress refining the in vitro creation of photoreceptors from iPSCs.

Second, Dr. Bernier and his team are using iPSCs to model retinal diseases and sequence their genetic structures. It is an old but reliable axiom in medical science that you must first understand a disease before you can treat it, and this is the guiding principle at play in this aspect of the work. By creating sheets of diseased photoreceptors in vitro and comparing these to healthy ones, they are able to better understand the genetic processes involved in the death of photoreceptors. In the world of stem cell therapy this knowledge is just as essential as the creation of new, functioning cells.

This two-pronged approach—the creation of healthy photoreceptors and the analysis of diseased ones—is designed to lead directly into clinical trials. With data gathered from this work, Dr. Bernier would like to launch a Phase 1 clinical trial to test the viability of stem cell transplantation in human patients. Such a trial is integral to the development of treatments; without clinical trials, discoveries from the lab would not make their way into the hands of those who need them.

That is the end-point of the team’s work: the creation and transplantation of healthy photoreceptors to restore vision in those who have lost it. Their work has been incredible, and your generosity can bring them to the finish line.

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